Newborn screening + enzyme replacement therapy = improved lysosomal storage disorder: outcomes in infantile-onset Pompe disease

J Pediatr. 2015 Apr;166(4):800-1. doi: 10.1016/j.jpeds.2014.12.028. Epub 2015 Jan 29.
No abstract available

Publication types

  • Editorial
  • Comment

MeSH terms

  • Female
  • Glycogen Storage Disease Type II / diagnosis*
  • Humans
  • Male
  • Neonatal Screening / methods*
  • alpha-Glucosidases / therapeutic use*

Substances

  • GAA protein, human
  • alpha-Glucosidases